In-development respiratory treatment has potential to save thousands of lives

UCLA doctor developing first targeted treatment of acute respiratory ailments.

When you work in pediatric critical care, you end up seeing a lot of children admitted to the hospital for respiratory problems — often acute lung injury or acute respiratory distress syndrome (ARDS).

And as you administer treatment to these patients, you’re limited to supportive measures, since as of now there is no targeted therapy that treats the underlying cause. This is the frustrating situation Andreas Schwingshackl, MD, PhD, finds himself in as the associate professor of pediatrics in the division of pediatric critical care medicine at UCLA.

“Despite over 50 years of intense basic science and clinical research, we do not have a single molecular target that translates into improved patient outcomes,” Dr. Schwingshackl says. “Everything we have done and are doing in the ICU to this day is supportive measures.”

Dr. Schwingshackl’s unique expertise — practicing pediatric medicine while researching age-agnostic respiratory pulmonology — is now focused on acute lung injury and ARDS in order to make the practice of using supportive measures without targeted treatments obsolete.

A massive unsolved problem

For acute lung injury and ARDS, statistics alone make the case that a targeted treatment is sorely needed.

The two ailments account for roughly 200,000 hospital beds for U.S. adults each year, with a 35 to 40% mortality rate that results in about 75,000 deaths. In children, the case number is around 8,000 per year and the mortality rate is between 15 and 30%, accounting for 1,400 deaths.

But Dr. Schwingshackl is on a mission to bring those shocking mortality numbers down.

“We are on the search for new therapeutic targets,” he says, “that ultimately can be translated into an improved patient outcome at the bedside.”

A potential breakthrough solution

Dr. Schwingshackl’s team is collaborating with a group of researchers from University of California San Francisco that develops compounds to target specific potassium channels in the body to decrease inflammation.

“We’re currently trying to find out if the targets we have, these potassium channels, are a good target to start with,” Dr. Schwingshackl says. “And at the same time, make sure that the compounds we’re using are not harmful to the body.”

Thus far in the treatment-development process, the results are encouraging: “No toxicity whatsoever.”

Ultimately, Dr. Schwingshackl says, these compounds could lead to a ventilator-administered drug that benefits acute lung injury and ARDS patients of all ages, since the electrophysiology of the lung is relatively consistent across children and adults.

A substantial impact

With the mice-focused phase of testing wrapping up soon, the next step is to begin testing on larger animals. After that, the team will begin collaborating with pharmaceutical companies to develop a market-ready drug.

The expected end result: a treatment that can double or triple the chances of survival and speed up recovery in patients who are moderately to severely ill with acute lung injury or ARDS.

For thousands and thousands of patients, “the impact could be substantial,” Dr. Schwingshackl says.

Learn more about UCLA Mattel Children's Hospital and the Children's Discovery and Innovation Institute.